Rectify Pharma Announces Positive Functional Modulator RTY-694 as Development Candidate for Primary Sclerosing Cholangitis

RTY-694 is a potent, orally active ABCB4/BSEP positive functional modulator (PFM) that improves bile composition and enhances bile flow

The differentiated mechanism of a dual-targeted ABCB4/BSEP PFM addresses the underlying pathophysiology of cholestasis and cholangitis associated with multiple hepatobiliary diseases, including primary sclerosing cholangitis

RTY-694 improved bile composition and demonstrated reductions in bile duct injury, cholestasis, inflammation and fibrosis

CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) — Rectify Pharmaceuticals, Inc., (“Rectify”) a biotechnology company developing positive functional modulators (PFMs), small molecule therapeutics that restore and enhance membrane protein function, today announced that RTY-694 was selected as the lead PFM for its hepatobiliary program and is advancing to first-in-human clinical trials for primary sclerosing cholangitis (PSC).

“Rectify was founded to harness the untapped therapeutic potential of restoring or enhancing membrane protein function to address the underlying drivers of disease. The selection of RTY-694 as our lead candidate for the treatment of PSC marks an important milestone for our company and validates our breakthrough proprietary PFM platform for developing therapies that restore and enhance membrane protein function,” said Rajesh Devraj, Ph.D., President and Chief Executive Officer of Rectify. “With this lead candidate nomination and a strong pipeline of earlier stage programs for cardio-renal-metabolic and neurodegenerative diseases, we are poised to establish PFMs as a novel small molecule modality and Rectify as the industry-leading PFM company.”

Pol Boudes, M.D., Chief Medical Officer of Rectify, added “We believe our differentiated dual-targeted approach of addressing ABCB4 and BSEP transporter dysfunction to improve bile composition and enhance bile flow has the potential to rectify the core pathophysiology of PSC and multiple hepatobiliary diseases. We look forward to advancing RTY-694 to first-in-human clinical trials as we pursue our mission of helping patients with serious diseases.”

PSC is a debilitating orphan bile duct disease characterized by inflammation, fibrosis, and narrowing of the medium and large intra- and extra-hepatic bile ducts that leads to cholangitis, cholestasis and ultimately liver failure. Patients living with PSC experience a high symptom burden that reduces their quality of life and often requires hospitalization. They also face a 400-fold higher rate of developing cholangiocarcinoma, a bile duct cancer, compared to the general population. Many patients will eventually require liver transplant due to disease progression and complications. There are currently no approved medications for the treatment of PSC.

RTY-694 is an orally acting dual-targeted PFM that addresses the core pathophysiology of PSC disease progression by increasing the function of the ABCB4 and BSEP transporters, two membrane proteins that play a critical role in maintaining bile composition and bile flow. In translational mouse models, RTY-694 demonstrated improvements in bile duct health, inflammation and fibrosis including:

• Increases in biliary phospholipids, a key bile component that buffers inflammatory free cholesterol and detergent bile acids

• Reductions in serum bile acids, ALT and ALP levels

• Improvements in ductular reaction characterized by a reduction in cholangiocyte proliferation and inflammation

• Reductions in TGF-β activation and periportal fibrosis

About Rectify Pharmaceuticals, Inc. (“Rectify”)

Rectify is advancing Positive Functional Modulators (PFMs), a novel class of oral, small molecules that restore and enhance membrane protein function to address the underlying cause of serious diseases. Rectify’s PFMs have potential to modulate the activity of wild-type and mutated membrane bound proteins, a historically difficult challenge with a small molecule approach. The Company’s breakthrough product platform enables efficient and rapid discovery of first- and best-in-class small molecule therapies with the potential to address membrane protein dysfunction for treatment of rare and common diseases, including liver, cardio-renal-metabolic, and neurodegenerative diseases. Rectify was founded and seeded by Atlas Venture who co-led the $100M Series A round with Omega Funds and were joined by Forbion and Longwood Fund.
For more information, please visit www.rectifypharma.com or follow us on X and LinkedIn.

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Michael Rubenstein
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